When the Huntington's community, charity and researchers come together for a heartwarming weekend.
It was amazing to have our conference held in person again after four years of online events. We managed to secure a wonderful venue, Crewe Hall which was beautiful, accessible and offered all that we needed to run a successful event. Thank you to our Headline Sponsors, Sage Therapeutics and Wave Life Sciences for making this event possible.
We opened the event with the mandatory Annual General Meeting, giving members of the charity the opportunity to hear about the finances and accounts and to vote for the trustees.
This was followed by the year in review from Cath Stanley, Huntington's Disease Association Chief Executive. It was affirmative to hear all the numbers and achievements of that charity over the last year in one presentation and discussing the impact that this has had on our service users and the wider community. Plans for the future were discussed in line with our recently launched five-year strategy and five key goals that we want to achieve as a charity.
Conference speakers
We were fortunate to be joined by some fantastic speakers over the course of the day. Between the talks, there was time for everyone to network whilst getting food and hot drinks as well as viewing the stands to gain further information from pharmaceutical companies and care homes.
Professor Ed Wild
Ed, Consultant Neurologist at National Hospital for Neurology and Neurosurgery started off the conference with an engaging update about many of the known and also unknown pharma companies and their clinical trials. His aim was to highlight the positive breakthroughs that are happening in Huntington's trials at the moment. Below are some of the key points.
PTC Therapeutics
Their pill-form drug, Votoplam, is known as a splicing drug which means it aims to modify the sequence attached to the gene so that the end of the sequence (the longer CAG repeat) is killed off by the cells. Votoplam is lowering both the mutant HTT gene (the damaged Huntingtin gene) and the wild-type HTT gene (the undamaged gene). After phase 2 of the trial, PTC Therapeutics had successfully lowered the HTT protein by 43%.
uniQure
The most invasive of trials but one that if successful would mean a one-time treatment. It involves six injections into the brain and is a high dose but they expect it to have a dramatic improvement. Multiple things are tracked during research trials, one of which is something called neurofilament, a biomarker that reflects the current health of the brain. Two years after the operation the neurofilament had risen due to the surgery but two years later they saw an 11% drop which has never been seen before in a Huntington's trail. We expect more news from this in mid 2025.
Skyhawk
This pharma company was under the radar due to the trials only taking place in Australia but it has not only completed trials, it is actually being used to treat patients in Australia. This drug is another splicer (lowering the Huntingtin protein by changing the sequence in the damaged Huntingtin gene). This drug has reduced the HTT (Huntingtin gene) by an incredible 72%.
The bad things are turning into good things and the good things are turning into great things. - Ed Wild
Sarah Gunn
Sarah's talk updated us on the Keeping Yourself in Mind: Psychological therapies for people affected by Huntington’s disease project that she has been working on as a collaboration with us. Sarah wanted to try something different to the off-the-shelf therapies that aren't always effective for people with Huntington's. Acceptance and Commitment Therapy is based on finding the person's values what matters to them and in her courses, she has found there to be up to a 50% reduction in low mood and anxiety.
Sarah is also a Lecturer in Clinical Psychology and she hosted a selection of lighting talks from her degree students:
- Suzanne Buswell (Research Assistant) – Exploring the experiences of Acceptance and Commitment Therapy for caregivers to those with Huntington's disease
- Paige Lindo (Trainee Clinical Psychologist) – Understanding how Huntington's affects the wellbeing and relationships of people with the disease, their families and caregivers
- Catherine Lyon (Trustee) – Experiences and perceptions of affected individuals, families, carers and healthcare professionals regarding end-of-life planning in Huntington's disease
Chris Leak
Chris is a Huntington's Disease Association ambassador who publicly speaks about his journey navigating Huntington's disease. Chris shared his story from first finding out about the disease and highlighting all the support he has had from the Huntington's Disease Association and his local Specialist Adviser, Rachel along the way, including support at work, joining support groups and holistic therapy sessions to having a platform where he could feel empowered to share his story. He included difficulties he has come across along the way as a person living with the disease but highlighted the importance of collaborative working between the lived experience and the charity. Chris received a well-deserved ongoing round of applause at the end.
Rachel Taylor
Rachel is a Clinical Nurse Consultant at the National Hospital for Neurology and Neurosurgery. Rachel's talk, how we support families affected by Huntington's started off with a family tree. Rachel went through the family tree covering the person with the disease, those at risk, those positive, negative and relatives and showed the types of support each individual may require. Her talk reflected back to Chris's lived experience throughout which really emphasised the importance of us working with the community in order to ensure those affected get the support that is right for them.
After the conference, there was more time to network with other attendees before everyone checked into their rooms to get ready ahead of the evening. Guests enjoyed a drinks reception followed by a three-course dinner and ending the night with a disco.
We would like to thank all of our community who joined us for the weekend as well as our speakers, event sponsors and those with stands. It was amazing to have everyone together in one room to look at both the achievements from the last year and also to look at the future of what we aim to achieve as a community. We would also like to thank Rikki Marr who did a live graphic of the conference throughout the day.
Event sponsor
This year's event was sponsored by Sage Therapeutics and Wave Life Sciences. We are very grateful for their dedication to Huntington's disease research and for helping make this event possible.
We also thank our other sponsors, Roche, UniQure, and Prilenia. Their contributions made this event possible.
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